AstraZeneca and Ionis are planning a US launch next month for Wainua, their treatment for polyneuropathy in life-threatening disease transthyretin-mediated amyloidosis (AT
Alnylam has abandoned plans to add cardiomyopathy to the label for transthyretin-mediated amyloidosis (ATTR) therapy Onpattro after the FDA turned down its marketing appli
Despite a sceptical appraisal by FDA reviewers, Alnylam's Onpattro has been recommended for approval as a treatment for transthyretin amyloidosis cardiomyopathy (ATTR-CM)
BridgeBio is getting ready to file for FDA approval of its acoramidis drug for transthyretin amyloidosis (ATTR) cardiomyopathy, armed with positive results in a phase 3 tr
Four years after getting the first drug approved by the FDA to treat rare disease hereditary ATTR amyloidosis, Alnylam has got the go-ahead for a second – Amvuttra – which has a simpler, mo
AstraZeneca has clearly made the rare disease transthyretin amyloidosis (ATTR) a key component of its rare disease pipeline, licensing a second drug candidate in the space of a couple of mo
Therapeutic or focused ultrasound began being applied to neurologic conditions less than a decade ago, but its potential in a wide spectrum of brain applications is high.
Investigator sites are essential for successful execution of clinical trials – ensuring studies are conducted ethically, safely, and in compliance with regulatory standards.